Eplontersen
Orphan Drug Cold Chain RequiredFDA Approved (2023), EMA Approved (2024)
Contents
Description
Eplontersen is a ligand-conjugated antisense oligonucleotide that reduces production of transthyretin (TTR) protein. It is indicated for hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) in adults. The drug offers self-administered subcutaneous dosing monthly, improving upon earlier therapies requiring more frequent administration.
Indications & Therapeutic Use
Hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN)
Global Availability (5 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Eplontersen
| Generic Name | eplontersen |
| Brands | 1 brand available |
| Active Ingredient | eplontersen sodium |
| Drug Class | Hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) |
| Manufacturer | Ionis Pharmaceuticals/AstraZeneca |
| Dosage Forms | Subcutaneous injection, 45mg/0.5mL pre-filled syringe |
| Medical Code | N07XX13 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 21 days |
| Reg. Status | FDA Approved (2023), EMA Approved (2024) |
| Clinical Trial | NCT04136184 |
| Countries | 5 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations6 Validated Nodes